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Study on AVB-101 for Patients with Frontotemporal Dementia with Progranulin Mutations

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Frontotemporal Dementia with Progranulin Mutations (FTD-GRN). This is a type of dementia that affects the frontal and temporal lobes of the brain, often leading to changes in personality, behavior, and language. The treatment being tested in this study is called AVB-101, which is a solution designed for injection or infusion. AVB-101 is a special type of therapy that uses a Recombinant AAV-9 vector to deliver the human progranulin protein directly into the brain. This is done through a procedure called intrathalamic infusion, which involves administering the treatment into a specific area of the brain known as the thalamus.

The purpose of this study is to evaluate the safety and initial effectiveness of AVB-101 in individuals with FTD-GRN. Participants in the study will receive a one-time administration of AVB-101, and their health will be monitored over a period of time to observe any changes or side effects. The study will take place over several years, with an initial follow-up period of 26 weeks and a total follow-up period of up to five years. During this time, researchers will keep track of any adverse events, changes in vital signs, and other health indicators to ensure the treatment is safe and to gather preliminary data on its potential benefits.

Throughout the study, participants will undergo various assessments, including MRI scans and laboratory tests, to monitor their condition and the effects of the treatment. The study aims to provide valuable information on the safety and potential benefits of AVB-101 for people with FTD-GRN, contributing to the development of new treatment options for this challenging condition.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    The initial assessment involves a comprehensive evaluation to confirm eligibility for the study. This includes a review of medical history, a physical examination, and specific tests such as magnetic resonance imaging (MRI) to measure thalamic volume.

    Eligibility criteria include being between 30 to 75 years old, having a confirmed genetic mutation related to frontotemporal dementia, and meeting specific clinical criteria for the condition.

  2. Step 2

    Treatment administration

    The treatment involves a one-time administration of AVB-101, a solution for injection or infusion, delivered directly into the brain through a procedure known as bilateral intrathalamic infusion.

    This step is designed to evaluate the safety and initial effectiveness of the treatment in individuals with frontotemporal dementia due to progranulin mutations.

  3. Step 3

    Initial follow-up period

    Following the treatment, there is a 26-week period of close monitoring. During this time, various health parameters are assessed, including vital signs, neurological examinations, and laboratory tests.

    The focus is on identifying any adverse effects, changes in mental status, and other health indicators.

  4. Step 4

    Long-term follow-up

    The study includes a long-term follow-up period lasting up to five years. This involves regular assessments to monitor the ongoing safety and potential benefits of the treatment.

    Key measures include changes in protein levels in the cerebrospinal fluid and blood, as well as evaluations of cognitive and functional status.

Who can join the trial?

10 criteria

  • Must be a male or female between 30 and 75 years old.
  • Must have a study partner who understands the study and can help with participation and assessments. This partner must also agree to participate if required.
  • Must carry a specific genetic change called a **granulin (GRN) mutation**, which is linked to the condition being studied. This must be confirmed by a genetic test approved by the study sponsor.
  • Must have **frontotemporal dementia (FTD)**, which is a type of dementia. This is shown by specific scores on tests used to assess dementia.
  • Must show signs of certain types of FTD, such as changes in behavior or language difficulties.
  • Must have a **thalamic volume** of at least 5.0 cubic centimeters on each side of the brain, as seen on an MRI scan. The thalamus is a part of the brain.
  • If a woman can have children, she must have a negative pregnancy test and not be breastfeeding before treatment.
  • If the participant or their partner can have children, they must agree to use a very effective method of birth control during the study.
  • Must be willing and able to follow all study procedures and attend all scheduled visits.
  • Must be able to give written consent to participate in the study and agree to appoint someone to make decisions on their behalf if they are unable to do so during the study.

Who cannot join the trial?

6 criteria

  • Individuals who do not have a confirmed diagnosis of Frontotemporal Dementia With Progranulin Mutations (FTD-GRN) cannot participate. This is a specific type of dementia related to genetic changes.
  • Participants must be within a certain age range, typically adults, to be eligible.
  • Both males and females can participate, but certain health conditions or factors may exclude someone.
  • People who are considered part of a vulnerable population may not be eligible. This term refers to groups who might be at higher risk of harm or exploitation, such as children, pregnant women, or those with certain disabilities.
  • Individuals with other medical conditions or taking certain medications that could interfere with the study may be excluded.
  • Participants must be able to understand and agree to the study requirements, which may exclude those with severe cognitive impairments.
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Investigated drugs

AVB-101 is an experimental treatment being studied for its safety and potential effectiveness in people with a specific type of dementia called Frontotemporal Dementia with Progranulin Mutations (FTD-GRN). This treatment is administered directly into the brain through a procedure known as bilateral intrathalamic infusion. The goal of this study is to determine if AVB-101 is safe and well-tolerated by patients, as well as to gather preliminary information on its potential benefits for this condition.

What is already known about the treatment

AVB-101 – AVB-101 is administered through a one-time bilateral intrathalamic infusion, specifically targeting the brain. It is currently in Phase 1/2 clinical trials, focusing on its safety and preliminary effectiveness. The main therapeutic indication for AVB-101 is Frontotemporal Dementia with Progranulin Mutations (FTD-GRN). At the molecular level, AVB-101 is designed to address the underlying genetic mutations associated with this form of dementia, potentially modifying disease progression. It falls under the pharmacological classification of gene therapy, aiming to correct or compensate for the genetic defects causing the condition.

Investigated diseases

Frontotemporal Dementia With Progranulin Mutations (FTD-GRN) – This is a type of dementia that primarily affects the frontal and temporal lobes of the brain, leading to changes in personality, behavior, and language. It is associated with mutations in the progranulin gene, which result in reduced levels of the progranulin protein. The disease typically begins with subtle changes in behavior and personality, such as apathy, disinhibition, or compulsive behavior. As it progresses, individuals may experience difficulties with language, including speaking and understanding words. Over time, the disease can lead to more severe cognitive impairments, affecting memory and executive functions. The progression of symptoms can vary widely among individuals, with some experiencing rapid changes and others having a more gradual decline.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2023-509444-10-00Protocol codeAVB-PGRN-001Estimated enrolment36 patientsSponsorAviadobio Limited

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